FDA approves treatment for rapidly progressing type of cancer

The US Food and Drug Administration (FDA) has approved a targeted therapy, Besponsa (inotuzumab ozogamicin), for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

“For adult patients with B-cell ALL whose cancer has not responded to initial treatment or has returned after treatment, life expectancy is typically low,” said Dr Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “These patients have few treatments available and today’s approval provides a new, targeted treatment option.”

This form of cancer is rapidly progressing and affects the bone marrow with an over production of B-cell lymphocytes. It is thought that Besponsa (from Pfizer) works by binding to the B-cell ALL cancer cells, blocking growth.

Safety and efficacy of the targeted therapy has been evaluated in a randomised trial of 326 patients, who had received one or two prior treatments. Patients either received treatment with Besponsa or an alternative chemotherapy regimen and then the proportion of patients who had no evidence of disease or complete remission after treatment was assessed. Out of the 326 patients, 218 were evaluated of whom 35.8% who were given Besponsa experienced complete remission for around 8 months and of those receiving the alternative chemotherapy treatment, 17.4% experienced complete remission for around 4.9 months.

Common side effects of Besponsa include low levels of platelets (thrombocytopenia), low levels of certain white blood cells (neutropenia, leukopenia), infection, low levels of red blood cells (anaemia), fatigue, severe bleeding (haemorrhage), fever (pyrexia), nausea, headache, low levels of white blood cells with fever (febrile neutropenia), liver damage (transaminases and/or gamma-glutamyltransferase increased), abdominal pain and high levels of bilirubin in the blood (hyperbilirubinemia).